Core Outcome Set for Paediatric Achalasia

Achalasia is a rare disease of oesophageal dysmotility, affecting 0.11 - 1.80 per 100,000 children. Diagnosis is often delayed and there is considerable variation in investigation and treatment of these children. There is a paucity of evidence regarding the optimal management of paediatric achalasia. Outcomes important to patients are frequently omitted or are inconsistently reported, which limits the value of such studies for informing practice and policy. We aim to develop a COS for paediatric achalasia, involving European stakeholders, that can be used in future research studies.

Contributors

Jonathan J Neville, Iris den Uijl, Willemijn Irvine, Simon Eaton, Frederic Gottrand, Nigel J Hall, ERNICA, EUPSA, ESPHGAN

Further Study Information

Current Stage: Ongoing
Date: September 2024 - August 2025
Funding source(s): TBC


Health Area

Disease Category: Child health

Disease Name: Achalasia

Target Population

Age Range: 16

Sex: Either

Nature of Intervention: Drug, Surgery

Stakeholders Involved

- Charities
- Clinical experts
- Families
- Patient/ support group representatives
- Researchers
- Service users

Study Type

- COS for clinical trials or clinical research
- COS for practice

Method(s)

- Consensus meeting
- Delphi process
- Focus group(s)
- Literature review

Outcomes will initially be identified from systematic reviews of the literature and stakeholders, including paediatric and adult surgeons and gastroenterologists, patient groups and allied health professionals. An online Delphi consensus exercise will then be undertaken by the same stakeholders. All outcomes will be rated on a 9-point Likert scale over three rounds to reach consensus. All rated outcomes will then be discussed at a consensus meeting. Study findings will be disseminated through research networks, presentations at relevant conferences and peer-reviewed publications. Results will also be reported to patient groups.